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更新于：2026-11-03
NI-006
更新于：2026-11-03
概要
基本信息
原研机构 |
最高研发阶段临床3期 |
首次获批日期- |
最高研发阶段(中国)临床3期 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (日本) |
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结构/序列
Sequence Code 1242171075L
来源: *****
Sequence Code 1242171076H
来源: *****
关联
8
项与 NI-006 相关的临床试验NCT07789483
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
NCT07608354
A Phase IIb, Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo in Adult Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
NCT07213583
A Phase 2, Single Arm, Multicenter Study to Evaluate the Pharmacodynamics and Safety of Re-Treatment With ALXN2220 in Patients With Transthyretin Amyloid Cardiomyopathy
100 项与 NI-006 相关的临床结果
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100 项与 NI-006 相关的转化医学
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100 项与 NI-006 相关的专利（医药）
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7
项与 NI-006 相关的文献（医药）2025-03-01CURRENT OPINION IN CARDIOLOGY
New therapies to treat cardiac amyloidosis
Review
作者: Ahmad Masri ; Daniel Kamna ; Olives Nguyen
Purpose of review:
Review advancements in therapies for transthyretin (ATTR-CM) and immunoglobulin light chain (AL-CM) cardiac amyloidosis.
Recent findings:
In ATTR-CM, tafamidis remains the cornerstone therapy, with Food and Drug Administration (FDA) approval for over 5 years. Acoramidis, another transthyretin stabilizer, has very recently been FDA-approved following positive results in the ATTRibute-CM trial. Vutrisiran, a transthyretin gene silencer, demonstrated efficacy in the HELIOS-B trial and awaits FDA review. Eplontersen's CARDIO-TTRansform trial, the largest ATTR-CM study to date, is expected to report by late 2025. Innovative approaches such as NTLA-2001 (a CRISPR-Cas9 therapy) and fibril depleters like ALXN2220 and coramitug are advancing in clinical trials. In AL-CM, daratumumab, cyclophosphamide, bortezomib, and dexamethasone (Dara-CyBorD) has established itself as the standard of care. Novel antiplasma cell therapies include CAR-T cells and bispecific antibodies (teclistimab) and fibril depleters. Birtamimab improved survival in advanced AL-CM during the VITAL trial and is under investigation in AFFIRM-AL. Anselamimab is in phase III CARES trials, whereas AT-02 undergoes early-phase testing for ATTR-CM and AL-CM.
Summary:
The therapeutic landscape for ATTR-CM and AL-CM is rapidly evolving, driven by novel therapies targeting diverse mechanisms. Ongoing clinical trials promise to further refine the standard of care and improve outcomes for patients with cardiac amyloidosis.
2025-01-01CLINICAL PHARMACOLOGY & THERAPEUTICS
Prediction of Cardiac ATTR Depletion by NI006 (ALXN2220 ) Using Mechanistic PK /PD Modeling
Article
作者: Michalon, Aubin ; Machacek, Matthias ; Cortijo, Cédric ; Grimm, Jan ; Nitsch, Roger M. ; Buller, Fabian ; Mercuri, Michele F. ; Hock, Christoph ; Kahr, Peter C. ; Renaud, Lionel ; Udata, Chandrasekhar
NI006 (aka ALXN2220) is a therapeutic antibody candidate in phase III clinical development for the depletion of amyloid transthyretin (ATTR) in patients with ATTR cardiomyopathy, an infiltrative cardiomyopathy leading to increased left ventricular wall thickness (LVWT). The mode‐of‐action consists in removal of disease‐causing amyloid accumulations by activating phagocytic immune cells, a mechanism without precedent in cardiology. To select a safe and potentially efficacious dose range and treatment duration for a combined first‐in‐human and proof‐of‐concept clinical phase Ib study, we developed a mechanistic pharmacokinetic and pharmacodynamic (PK/PD) model that can predict NI006 exposure, its effects on cardiac amyloid load and on LWVT, which is a predictor of heart failure in this disease. The PK/PD model predictions supported 0.3 mg/kg monthly dosing as a safe starting dose and identified 10–60 mg/kg monthly as the potentially efficacious dose range with substantial and dose dependent cardiac amyloid burden reduction within 4 months for 60 mg/kg and 10 months for 10 mg/kg. These predictions were in good agreement with the observed primary results of the clinical phase Ib study where amyloid burden was measured by imaging. This novel translational PK/PD model provided important predictions to guide the design of the phase Ib study of NI006, indicating the value of this approach to integrate preclinical results into clinical trial design and increase translational success.
2024-09-01Current opinion in cardiology
Treatment of transthyretin cardiac amyloidosis
Review
作者: Abdirahman Wardhere ; Mathew S. Maurer ; Dimitrios Bampatsias
Purpose of review:
Tafamidis is currently the only approved disease-modifying treatment for ATTR-CM. However, there have been important developments in the treatment of ATTR-CM, as the results of two phase 3 trials were published and several other trials are in their final stages. In this review, we summarize current and future therapies for ATTR-CM.
Recent findings:
Recently, acoramidis, a TTR stabilizer has been proven to be effective in reducing mortality and morbidity compared to placebo in the ATTRibute-CM trial. Additionally, patisiran, an RNA silencer, preserved functional capacity and quality of life compared to placebo in the APOLLO-B trial. However, the FDA declined to approve patisiran for ATTR-CM. The results of phase 1 trial of ALXN2220, an antiamyloid antibody raise hope for reversal of myocardial damage by amyloid depletion. Phase 3 trials evaluating the efficacy of different RNA silencers, gene editing with CRISPR-Cas9, and other anti-amyloid antibodies are ongoing.
Summary:
Therapies targeting different mechanism in the pathophysiology of ATTR-CM provide new alternatives for treating patients with ATTR-CM. Future research should focus on comparing their effectiveness, the potential of combined treatment with agents from different classes and on identifying the patients who will benefit most from each class of medication.
100 项与 NI-006 相关的药物交易
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外链
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| D13260 | - | - | - |
研发状态
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 美国 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 日本 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 阿根廷 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 澳大利亚 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 奥地利 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 比利时 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 巴西 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 加拿大 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 捷克 | 2024-01-11 | |
| 遗传性转甲状腺素蛋白淀粉样变性 | 临床3期 | 丹麦 | 2024-01-11 |
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临床结果
临床结果
适应症
分期
评价
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临床1期 | 46 | 顧廠構鹹顧繭鹽齋糧網(糧鏇衊選構蓋壓鏇選夢) = 憲醖蓋築築築選憲窪淵 壓獵齋網構積築夢蓋製 (願醖憲糧築夢願網膚夢, 54 ~ 63) 更多 | 积极 | 2026-08-30 | |||
临床1期 | 23 | 鹹廠製餘齋壓襯遞獵廠(淵鹹顧糧繭鑰遞築繭鹽) = 鬱壓鬱衊襯衊艱網築觸 鏇遞鑰繭壓餘選積襯糧 (製衊網艱製積積積選廠 ) | 积极 | 2026-06-26 | |||
临床1期 | 40 | 積遞築築鹹廠製構製簾(鹹製鑰網構艱壓夢憲鹹) = The use of NI006 was associated with no apparent drug-related serious adverse events. 構蓋觸繭鏇夢鬱衊糧顧 (製獵夢齋艱選選壓網鹹 ) | 积极 | 2023-07-30 | |||
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